Ipsen Wins Approval for FOP Drug in Canada

Health Canada approved Ipsen’s Sohonos to reduce the formation of new bone outside the skeletal system in adults and children (females aged 8 years and above and males 10 years and above) with the rare bone condtion fibrodysplasia ossificans progressive.

Photo: Howard Mayer, executive vice president and head of research and development for Ipsen

The decision is the first approval for Sohonos worldwide.

Fibrodysplasia ossificans progressive (FOP) is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification, which can be preceded by painful soft tissue swelling or “flare-ups.” Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy. It is an ultra-rare genetic disorder.

Sohonos (palovarotene) is an oral, selective RARγ agonist developed as a treatment for people living with the debilitating ultra-rare genetic disorder, FOP. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. It is a member of the retinoid class of drugs that is associated with birth defects in humans.

Sohonos must not be used by patients who are, or intend to become, pregnant due to the risk of teratogenicity. To minimize fetal exposure, Sohonos is to be administered only if all conditions for pregnancy prevention are met. Sohonos has been shown to cause premature physeal closure in growing children with FOP and periodic monitoring is recommended.

“FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families,” said Howard Mayer, executive vice president and head of research and development for Ipsen. “Until today, there was no approved medicine.”

As part of the ongoing commitment to the FOP and rare disease community, Ipsen plans to file in the United States in the first half of 2022 and is in discussions with other regulatory authorities around the world.

The approval is based on data from the ongoing MOVE trial, the first global multi-center phase 3 trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP.

Author: Rare Daily Staff